12.6.22, Pediatric Grand Rounds, " Transformative Treatment of Rare Disease - X-linked hypophosphatemia through the life span", Drs. Nancy Dunbar & Carolyn Macica

Relate the primary comorbid features of adult X-linked hypophosphatemia (XLH) to physical function.List and compare the Phase 3 Primary and Secondary endpoints for adults with XLH treated with Burosumab.Distinguish x-linked hypophosphatemic rickets from acquired forms of rickets such as vitamin D deficient rickets.Explain the mechanism of action of burosumab - a human recombinant anti-FGF-23 antibody therapy for X-linked hypophosphatemia.